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Trackers Clinical trials Leber Congenital Amaurosis
Clinical trial register · United States · industry-sponsored

Leber Congenital Amaurosis Trials in the USA

Every industry-sponsored study on the ClinicalTrials.gov registry that lists a US site and is open, about to open, active-but-closed, or enrolling by invitation. This deliberately excludes academic and other non-industry sponsors โ€” the full US registry across all sponsors runs to over 22,000 recruiting studies alone.

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Source: ClinicalTrials.gov. Complete coverage of the industry-sponsored slice โ€” all 9109 matching records are in this view (non-industry sponsors are out of scope by design, not truncated). Synced 19 Sep 2026, 04:27 UTC
9109
Studies in scope
industry-sponsored, all four open statuses
4946
Recruiting now
open to new participants
2196
Lead sponsors
across recruiting studies
Top states
Pennsylvania 6 California 3 Iowa 3 Texas 3 Florida 2
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Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA-Associated Inherited Retinal Degeneration (LCA-IRD)
NCT07681778Leber Congenital Amaurosis, Leber Congenital Amaurosis (LCA)
Opus Genetics, Inc
Phase 1/2
10
Arizona, Pennsylvania, Texas
2026-09-01
Recruiting
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
NCT06891443Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders
Laboratoires Thea
Phase 3
32
California, Florida, Iowa, Minnesota, Pennsylvania
2025-06-04
Recruiting
Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
NCT05906953Leber Congenital Amaurosis, Inherited Retinal Diseases Caused by RPE65 Mutations
HuidaGene Therapeutics Co., Ltd.
Phase 1/2
20
California, Texas
2023-10-31
Active, closed
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
NCT05203939Retinitis Pigmentosa, Leber Congenital Amaurosis
Ocugen
Phase 1/2
22
Arizona, California, Florida, Georgia, Oregon, Pennsylvania, Texas
2022-01-24
Active, closed
Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D
NCT03920007Leber Congenital Amaurosis, LCA, LCA1
Atsena Therapeutics Inc.
Phase 1/2
15
Oregon, Pennsylvania
2019-10-22
Active, closed
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
NCT03913143Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders
ProQR Therapeutics
Phase 2/3
36
Iowa
2019-04-04
Active, closed
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
NCT00999609Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Spark Therapeutics, Inc.
Phase 3
31
Iowa, Pennsylvania
2012-10
Active, closed
Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2
NCT01208389Leber Congenital Amaurosis
Spark Therapeutics, Inc.
Phase 1/2
12
Pennsylvania
2010-11
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Cite this view

Priya Life Science (2026). Clinical trial register: industry-sponsored studies with a US investigative site, filtered. Derived from ClinicalTrials.gov. Retrieved 19 September 2026. https://priyalifescience.com/clinical-trials-usa/condition/leber-congenital-amaurosis

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Listing here is informational and not medical advice; eligibility is determined by each study team. Compare pay for the teams running these studies in the US Pharma Salary Guide, see who's behind them in the US Company Directory, or track approvals on the FDA Approvals Tracker. For Irish, UK and EU sites, see the Ireland, UK and EU registers.